18 and older, any sex, with Beta Thalassemia Major or Beta Thalassemia Intermedia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Potential Recommended Dose as Determined by Number of Participants Experiencing Dose-Limiting Toxicities and Recommended DosePrimary· From first dose up to 28 days post the first dose
Number of participants with dose-limiting toxicities (DLT) are used to determine the potential recommended dose (PRD). PRD is defined as the highest dose with up to 1 out of 6 patients experiencing a DLT. DLT is defined as any side effects of the study treatment serious enough to prevent an increase in dose or level of treatment, including at least one of the following: Hypertension ≥ Grade 3 per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0; Hgb \> 14 g/dL sustained for four weeks; any NCI CTCAE toxicity ≥ Grade 3. Grade 3 is defined as sev
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
0
Dose Level 2: 0.5 mg/kg
0
Dose Level 3: 0.75 mg/kg
0
Dose Level 4: 1 mg/kg
0
Number of Participants With Red Blood Cell Transfusion Burden Reduction From Baseline During TreatmentSecondary· From baseline to the last dose of study treatment (up to approximately 112 months)
Transfusion burden at baseline is defined as the total number of units of RBC transfusions that participants received within 168 days (24 weeks) prior to the first dose of study therapy. Transfusion burden during treatment is defined as the total number of RBC transfusion units that each participant received during the treatment divided by the treatment duration and multiplied by 168 days. The result is a 168-day transfusion burden average. Baseline measurement includes RBC transfusion history for transfusion dependent and non-transfusion dependent participants, starting at 168 days prior to e
≥ 25% reduction
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
0
Dose Level 2: 0.5 mg/kg
2
Dose Level 3: 0.75 mg/kg
4
Dose Level 4: 1 mg/kg
3
≥ 33% reduction
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
0
Dose Level 2: 0.5 mg/kg
2
Dose Level 3: 0.75 mg/kg
3
Dose Level 4: 1 mg/kg
1
≥ 50% reduction
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
0
Dose Level 2: 0.5 mg/kg
1
Dose Level 3: 0.75 mg/kg
2
Dose Level 4: 1 mg/kg
0
Number of Participants With Hemoglobin Level Increase From Baseline in Non-Transfusion Dependent B-Thalassemia Intermedia ParticipantsSecondary· Measurements were taken in 9 and 12-week intervals, from baseline up to approximately 112 months
The Number of participants with a change in Hemoglobin levels will be listed for non-RBC transfusion dependent participants. Baseline assessments are the average of the last two measurements prior to the start of therapy.
9-week episode with Hgb ≥ 1.0 g/dl change from baseline (non-transfusion dependent participants)
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
5
Dose Level 2: 0.5 mg/kg
4
Dose Level 3: 0.75 mg/kg
6
Dose Level 4: 1 mg/kg
2
9-week episode with Hgb ≥ 1.5 g/dl change from baseline (non-transfusion dependent participants)
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
2
Dose Level 2: 0.5 mg/kg
2
Dose Level 3: 0.75 mg/kg
6
Dose Level 4: 1 mg/kg
1
12-Week episode with Hgb ≥ 1.0 g/dl change from baseline (non-transfusion dependent participants)
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
4
Dose Level 2: 0.5 mg/kg
4
Dose Level 3: 0.75 mg/kg
6
Dose Level 4: 1 mg/kg
1
12-Week episode with Hgb ≥ 1.5 g/dl change from baseline (non-transfusion dependent participants)
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
2
Dose Level 2: 0.5 mg/kg
2
Dose Level 3: 0.75 mg/kg
6
Dose Level 4: 1 mg/kg
1
Number of Participants Experiencing Adverse Events (AEs)Secondary· From first dose up to 112 days after the last dose of study treatment (up to 115 months)
An Adverse Event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a clinical investigation participant administered study treatment and that does not necessarily have a causal relationship with this treatment. Adverse events are graded on a scale from 1 to 5, with Grade 1 being mild and asymptomatic; Grade 2 is moderate requiring minimal, local or noninvasive intervention; Grade 3 is severe or medically significant but not immediately life-threatening; Grade 4 events are usually severe enough to require hospitalization; Grade 5 events a
Participants with at least one TEAE
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
8
Dose Level 1b: 0.3 mg/kg
9
Dose Level 2: 0.5 mg/kg
8
Dose Level 3: 0.75 mg/kg
12
Dose Level 4: 1 mg/kg
9
TEAE leading to dose interruption
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
2
Dose Level 1b: 0.3 mg/kg
5
Dose Level 2: 0.5 mg/kg
4
Dose Level 3: 0.75 mg/kg
2
Dose Level 4: 1 mg/kg
5
Participants with at least one serious TEAE
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
3
Dose Level 1b: 0.3 mg/kg
3
Dose Level 2: 0.5 mg/kg
3
Dose Level 3: 0.75 mg/kg
3
Dose Level 4: 1 mg/kg
3
Participants with at least one grade 2/3/4 TEAE
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
7
Dose Level 1b: 0.3 mg/kg
9
Dose Level 2: 0.5 mg/kg
6
Dose Level 3: 0.75 mg/kg
11
Dose Level 4: 1 mg/kg
9
Participants with at least one drug-related grade 2/3/4 TEAE
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
4
Dose Level 1b: 0.3 mg/kg
3
Dose Level 2: 0.5 mg/kg
4
Dose Level 3: 0.75 mg/kg
7
Dose Level 4: 1 mg/kg
7
Participants with at least one serious drug- related TEAE
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
2
Dose Level 1b: 0.3 mg/kg
1
Dose Level 2: 0.5 mg/kg
1
Dose Level 3: 0.75 mg/kg
0
Dose Level 4: 1 mg/kg
1
Participants with at least one TEAE leading to sotatercept withdrawal
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
3
Dose Level 1b: 0.3 mg/kg
0
Dose Level 2: 0.5 mg/kg
2
Dose Level 3: 0.75 mg/kg
3
Dose Level 4: 1 mg/kg
5
Concentrations of Sotatercept in SerumSecondary· Dose 1, Day 8; Dose 1, Day 15; Dose 2, Day 1; Dose 2, Day 8; Dose 3, Day 1; Dose 3, Day 8; Dose 4, Day 1; Dose 5, Day 1; Dose 6, Day 1
Sotatercept was administered as a subcutaneous injection every 21 days during the Treatment Period. Pharmacokinetic (PK) samples were collected at the pre-specified timepoints.
Dose 1, Day 8
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
598.3
289.2 – 1345.4
Dose Level 1b: 0.3 mg/kg
1454.05
1006.6 – 2768.3
Dose Level 2: 0.5 mg/kg
3045.45
1586.7 – 4480
Dose Level 3: 0.75 mg/kg
5837.65
3238.1 – 10590.5
Dose Level 4: 1 mg/kg
3874
2264.9 – 7003
Dose 1, Day 15
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
388.7
98.4 – 1046
Dose Level 1b: 0.3 mg/kg
1150.1
550.3 – 2158.4
Dose Level 2: 0.5 mg/kg
2329.75
952 – 3005.5
Dose Level 3: 0.75 mg/kg
4279.65
2107.8 – 6692
Dose Level 4: 1 mg/kg
2405.6
1825.3 – 4605.5
Dose 2, Day 1
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
294.75
112.9 – 547.1
Dose Level 1b: 0.3 mg/kg
770.3
317.2 – 1300.1
Dose Level 2: 0.5 mg/kg
1468.05
682 – 2550.5
Dose Level 3: 0.75 mg/kg
2955.2
1257.5 – 4450.8
Dose Level 4: 1 mg/kg
1701.5
888.6 – 4267.9
Dose 2, Day 8
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
683.1
250.1 – 1210.6
Dose Level 1b: 0.3 mg/kg
2065
1363.7 – 3626.4
Dose Level 2: 0.5 mg/kg
4136.8
1661.9 – 5573.8
Dose Level 3: 0.75 mg/kg
7753.35
5046.4 – 8805.6
Dose Level 4: 1 mg/kg
5148.1
3537.4 – 10418.7
Dose 3, Day 1
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
325.35
228.8 – 790
Dose Level 1b: 0.3 mg/kg
956.8
380.6 – 1552.6
Dose Level 2: 0.5 mg/kg
2157.3
422 – 3088.4
Dose Level 3: 0.75 mg/kg
3628.45
653.5 – 6429.1
Dose Level 4: 1 mg/kg
2602.6
822.2 – 5668.1
Dose 3, Day 8
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
758.1
444 – 1232
Dose Level 1b: 0.3 mg/kg
2319.9
1475.6 – 3667.4
Dose Level 2: 0.5 mg/kg
4507.3
2077.8 – 5762.3
Dose Level 3: 0.75 mg/kg
8373.05
5345.5 – 12177.8
Dose Level 4: 1 mg/kg
6050.8
3892.5 – 12139.6
Dose 4, Day 1
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
334.85
298 – 1014.9
Dose Level 1b: 0.3 mg/kg
1022.3
348.8 – 2096.9
Dose Level 2: 0.5 mg/kg
2441.6
784 – 2845.3
Dose Level 3: 0.75 mg/kg
4137.1
1447.1 – 6126.6
Dose Level 4: 1 mg/kg
2869.2
1478.9 – 4787.7
Dose 5, Day 1
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
385.1
287.5 – 467.5
Dose Level 1b: 0.3 mg/kg
1473.9
762.1 – 2764.4
Dose Level 2: 0.5 mg/kg
1618.8
953.4 – 3440
Dose Level 3: 0.75 mg/kg
4449.7
2340.4 – 5887.8
Dose Level 4: 1 mg/kg
3048
1125.5 – 6529.3
Number of Participants With Anti-Drug Antibody (ADA)Secondary· From first dose up to 4 months after last dose (up to approximately 116 months)
The number of participants with Anti-Sotatercept Antibody is a summary of antidrug antibody (ADA) status for ADA-evaluated participants. A participant is counted as 'positive' if there is any positive result captured during the study, a participant is counted as 'negative' if there is no positive result captured during the study. ADA data was collected Day 1 in dose schedules 1 through 6. Starting from Dose 7, ADA was measured at Day 1 every 3 Doses, then finally at the post-treatment follow-up visit at Month 2 and Month 4.
Negative
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
8
Dose Level 1b: 0.3 mg/kg
8
Dose Level 2: 0.5 mg/kg
8
Dose Level 3: 0.75 mg/kg
10
Dose Level 4: 1 mg/kg
9
Positive
Group
Value
95% CI
Dose Level 1a: 0.1 mg/kg
0
Dose Level 1b: 0.3 mg/kg
1
Dose Level 2: 0.5 mg/kg
0
Dose Level 3: 0.75 mg/kg
2
Dose Level 4: 1 mg/kg
0
Adverse events — posted to ClinicalTrials.gov
Time frame: Participants were assessed for all-cause mortality from their enrollment to study completion, (up to approximately 115 months). SAEs and Other AEs were assessed from first dose to 112 days following last dose (up to approximately 115 months).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Dose Level 1a: 0.1 mg/kg
Serious: 3/8 (38%)
Deaths: 0/8
Dose Level 1b: 0.3 mg/kg
Serious: 3/9 (33%)
Deaths: 0/9
Dose Level 2: 0.5 mg/kg
Serious: 3/8 (38%)
Deaths: 0/8
Dose Level 3: 0.75 mg/kg
Serious: 3/12 (25%)
Deaths: 0/12
Dose Level 4: 1 mg/kg
Serious: 3/9 (33%)
Deaths: 0/9
Serious adverse events (29 terms)
Reaction
System
Dose Level 1a: 0.1 mg/kg
Dose Level 1b: 0.3 mg/kg
Dose Level 2: 0.5 mg/kg
Dose Level 3: 0.75 mg/kg
Dose Level 4: 1 mg/kg
ANAEMIA
Blood and lymphatic system disorders
—
—
—
—
—
EXTRAMEDULLARY HAEMOPOIESIS
Blood and lymphatic system disorders
—
—
—
—
—
SPLENIC INFARCTION
Blood and lymphatic system disorders
—
—
—
—
—
PERICARDIAL EFFUSION
Cardiac disorders
—
—
—
—
—
ABDOMINAL PAIN UPPER
Gastrointestinal disorders
—
—
—
—
—
SUBILEUS
Gastrointestinal disorders
—
—
—
—
—
FATIGUE
General disorders
—
—
—
—
—
LOCAL SWELLING
General disorders
—
—
—
—
—
PYREXIA
General disorders
—
—
—
—
—
ANAPHYLACTIC REACTION
Immune system disorders
—
—
—
—
—
BACTERIAL PROSTATITIS
Infections and infestations
—
—
—
—
—
CORONA VIRUS INFECTION
Infections and infestations
—
—
—
—
—
LOWER RESPIRATORY TRACT INFECTION
Infections and infestations
—
—
—
—
—
PHARYNGOTONSILLITIS
Infections and infestations
—
—
—
—
—
SUBCUTANEOUS ABSCESS
Infections and infestations
—
—
—
—
—
FALL
Injury, poisoning and procedural complications
—
—
—
—
—
FOREARM FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
LUMBAR VERTEBRAL FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
PELVIC FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
PUBIS FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
ULNA FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
UPPER LIMB FRACTURE
Injury, poisoning and procedural complications
—
—
—
—
—
BONE PAIN
Musculoskeletal and connective tissue disorders
—
—
—
—
—
MUSCULAR WEAKNESS
Musculoskeletal and connective tissue disorders
—
—
—
—
—
HEPATIC NEOPLASM
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
—
—
—
Other adverse events (368 terms — click to expand)
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Celgene
Last refreshed: 18 June 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01571635.